I have been accepted to the clinical trial RTA 408. Although, I - TopicsExpress

I have been accepted to the clinical trial RTA 408. Although, I would like to be transferred from Philadelphia one hospital taking part in this clinical trial to UCLA in Los Angeles in California another hospital taking part due to the expenses incurred in flying back and forth numerous times in a Five month time. The pharmaceutical company is paying for the majority of my trips, but being closer to WA will be cheaper. Here is some info on this drug. feel free to ask any and all questions or concerns. RTA 408 works by inducing Nrf2, which regulates multiple genes that play both direct and indirect roles in the production of cellular energy (i.e., adenosine triphospha5 month span te or ATP) within the mitochondria. Directly, activation of the Nrf2 pathway increases the efficient use of fuel (fatty acids and glucose) by mitochondria and increases mitochondrial biogenesis and basal oxygen consumption. Indirectly, activation of Nrf2, through its antioxidative effects, balances reducing equivalents and maintains mitochondrial homeostasis and efficiency. In addition to its positive effects on metabolic efficiency, Nrf2 activation has been shown in preclinical studies to promote muscle repair and recovery and reduce markers of oxidative stress and muscle injury. Our collaborators and we have shown in preclinical studies that genetic or pharmacologic Nrf2 activation positively regulates mitochondrial function and energy production. We hope to translate this effect into improved physical functioning and reduced fatigue in patients with Friedreichs ataxia and mitochondrial myopathies. These rare, debilitating diseases currently have no approved therapies, noted Dr. Colin Meyer, Reatas Chief Medical Officer. The two initial Phase 2 trials will both be multi-center, double-blind, randomized, dose-ranging, placebo-controlled studies. The primary efficacy endpoint in both studies will be peak work as assessed during exercise testing. The studies will also explore changes in other measures of physical activity, fatigue, and biomarkers associated with mitochondrial functioning.

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